Pathogenic variants in the leucine-rich repeat kinase 2 (LRRK2) gene are linked to increased risk for Parkinson’s disease (PD), with G2019S being the principal variant. In the current study, researchers from Escape Bio Inc. presented the discovery and preclinical characterization of novel inhibitors of G2019S mutant-LRRK2 as potential therapeutic candidates for the treatment of PD.
Amyotrophic lateral sclerosis (ALS) is a disorder that leads to progressive muscle weakness and loss of muscle control due to affectation of the motor neurons. Increasing evidence points to defects in the nuclear envelope, which leads to disease progression.
Shanghai Institute of Materia Medica of the Chinese Academy of Sciences has described sodium channel protein type 10 subunit α (SCN10A; Nav1.8) blockers reported to be useful for the treatment of pain.
Saniona AB has initiated the candidate selection phase with a proprietary subtype selective frontrunner molecule from its Kv7 lead optimization program for epilepsy.
Researchers from The Brigham and Women's Hospital presented data from a study that aimed to evaluate the role of somatic variants in drug-resistant mesial temporal lobe epilepsy (MTLE). High-coverage whole-exome sequencing was conducted using DNA samples derived from the hippocampus and paired brain tissue and/or blood samples.
Mira Pharmaceuticals Inc. has entered into a licensing agreement with Miralogx LLC for exclusive rights in the U.S., Canada and Mexico to develop and commercialize Ketamir-2, a novel oral ketamine analogue for depressive disorder treatments.
Researchers from New York University College of Dentistry’s Pain Research Center in collaboration with the University of Pittsburgh found a candidate for chronic pain treatment among a library of 27 million compounds.
The Charcot-Marie-Tooth Association (CMTA) has participated in a seed extension round for Armatus Bio Inc., an emerging biotechnology company that is advancing a unique gene therapy clinical candidate to target Charcot-Marie-Tooth (CMT) type 1A.
Novel approaches are needed to improve anti-inflammatory drug delivery to the brain, particularly to glial cells, in order to reduce neuroinflammation.
Gilgamesh Pharmaceuticals Inc. has described pyridine derivatives 5-HT2A receptor agonists reported to be useful for the treatment of depression, anxiety disorders, and substance abuse and dependency.